PB2060: THE RESULTS OF A FOUR YEARS LONG OBSERVATION OF APLASTIC AMENIA PATIENTS DEPENDING ON A PROGRAM OF IMMUNOSUPPRESSIVE THERAPY
Authors: Daria Chebykina, Sergey Voloshin, Marina Zenina, Elene Shilova
Journal: HemaSphere (2023)

Topic: 12. Bone marrow failure syndromes incl. PNH - Clinical Background: Anti-thymocyte globulin (ATG) and cyclosporine A (CsA) that allow 60-70% of patients to achieve stable remission remain the preparations of the program of combined IST. However, the main disadvantages while undergoing IST are incomplete hematopoietic recovery, probability of clonal evolution and frequent recurrences. In real clinical practice the nature of treatment among some patients differs significantly from the recommended standards both because of poor tolerance of preparations that follows the pathology, and due to organizational issues. All of the above may decrease the efficiency of IST conducted. Aims: to evaluate the results of treatment of the aplastic anemia patients depending on IST program in the same center for four years. Methods: The results of 35 AA patients aged from 19 to 84 (median age 31) staying in FSBI Russian Scientific Research Institute of Hematology and Transfusiology of the Federal Medical and Biological Agency under constant control after the start of IST not less than 3 years over the period from 2012 to 2023 were analyzed. Severe AA (SAA) patients predominated – 18 (51%). PNH clone was detected in 16 patients (46%). The majority of the patients underwent IST 24 (69%). Immunosuppression in the regime of monotherapy was performed among 11 patients with moderate aplastic anemia (mAA): monotherapy ATG in 1 case (3%) and CsA in 10 cases (28%). A repeat course of ATG therapy was performed in 9 cases (25%) due to the clinical inefficiency. Results: Conducted IST turned out to be effective for 91% of patients, 12 patients (34%) achieved complete remission and 20 patients (57%) achieved partial remission. It is worth mentioning that 7 patients (23%) having achieved satisfactory treatment results had transient neutropenia for 3 years. After 4 years of observation CsA therapy was discontinued for 10 patients. 9% patients (n=3) didn’t respond to the treatment, two of them had Severe aplastic anemia (SAA). No lethal outcomes were observed. Two groups of patients were compared, group A of 15 patients (10 cases of SAA and 5 cases of MAA) that underwent standard full-dose IST, and group B of 20 patients (7 cases of SAA and 13 cases of MAA), some of whom for various reasons had declinations from generally accepted standards of treatment. When complete blood counts were compared, the average level of hemoglobin after 4 years of observation was 137,3 g/L in the group A and 117,4 g/L (p<0,01) in the group B, the level of platelets was 148,6 109/L in group A and 98,3 109/L (р<0,05) in group B, the level of neutrophils was 3,0 109/L и 1,7 109/L (p=0,03) accordingly. Speaking of the dynamics of recovery of the main blood components in the group A, after a year of therapy hemoglobin level of 33% of patients (n=5) increased, the level of platelets of 40% of patients (n=6) raised, 67% of patients (n=10) achieved a complete response to the therapy by the end of the fourth year of the treatment. As for the group B, 30% of patients (n=6) increased their hemoglobin, 25% (n=5) raised their level of platelets in 3 years, 30% (n=6) achieved a complete response to the therapy by the end of the fourth year of the treatment. Summary/Conclusion: Standard IST stays the most effective method of treatment AA. The received results demonstrate the obvious advantage of the established regimes of IST, meanwhile time and completeness of blood count recovery depend completely on the completeness of the provided treatment. Keywords: Cyclosporin A, ATG, Aplastic anemia, Immunosuppressive therapy